NICE Approves Zolgensma Gene Therapy for Pre-Symptomatic Treatment of SMA
The UK SMA Newborn Screening Alliance is delighted that Zolgensma™ (Onasemnogene abeparvovec) is now recommended as an option for treating presymptomatic SMA in babies aged 12 months and under, who have up to 3 copies of the SMN2 gene. Previously, babies diagnosed symptomatically could only access Zolgensma through a managed access agreement (MAA).
This recommendation is an important development in relation to newborn screening for SMA. The majority of babies diagnosed with SMA through newborn screening will not have symptoms of SMA. Treating pre-symptomatically is vital, as once symptoms of SMA have manifested, these cannot be reversed.
The UK National Screening Committee uses a list of 20 criteria to decide whether or not to recommend a new condition for the UK newborn screening programme. One criterion considers whether there is an effective intervention for patients identified through screening and “evidence that intervention at a pre-symptomatic phase leads to better outcomes.” NICE assesses clinical and cost effectiveness of treatments and considers pre-symptomatic treatment with Zolgensma to be clinically and cost effective, so this UK NSC criterion should now be met.
The UK NSC looks at newborn screening across the whole of the UK and this recommendation now aligns England with Scotland, where Zolgensma is already available routinely to pre-symptomatic infants. Wales and Northern Ireland normally follow NICE guidance.
Read more about the UK NSC criteria here and the Zolgensma NICE review here.